Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Videos

Hammerhead ribozymes for target validation.

John Goodchild1

  • 1Center for Discovery of Drugs and Diagnostics, University of Central Florida, 12722 Research Parkway, Orlando, FL 32826, USA. jgood@mail.ucf.edu

Expert Opinion on Therapeutic Targets
|September 12, 2002
PubMed
Summary

Hammerhead ribozymes offer a precise method for validating drug targets early in pharmaceutical development. These specific ribonucleases can selectively degrade target mRNA, potentially preventing costly late-stage failures.

Related Concept Videos

You might also read

Related Articles

Articles linked to this work by shared authors, journal, and citation graph.

Sort by
Same author

Therapeutic oligonucleotides.

Methods in molecular biology (Clifton, N.J.)·2011
Same author

A fight at the opera.

Current opinion in molecular therapeutics·2008
Same author

Oligonucleotide, antibody and peptide therapeutics--from design to the clinic.

Current opinion in molecular therapeutics·2004
Same author

Oligonucleotide therapeutics: 25 years agrowing.

Current opinion in molecular therapeutics·2004

Area of Science:

  • Molecular Biology
  • Biochemistry
  • Pharmaceutical Science

Background:

  • Pharmaceutical development faces high failure rates due to late-stage identification of ineffective drug targets.
  • Early-stage target validation is crucial to mitigate costly failures in drug development.
  • Hammerhead ribozymes present a potential solution for precise mRNA targeting and validation.

Purpose of the Study:

  • To review the mechanism, design, and application of hammerhead ribozymes for early drug target validation.
  • To compare hammerhead ribozymes with alternative methods like antisense oligonucleotides.
  • To provide information on the practical implementation of ribozymes in cellular and organismal models.

Main Methods:

  • Detailed examination of hammerhead ribozyme chemistry, cleavage specificity, and site selection.
  • Categorization of ribozymes into endogenous and exogenous systems.
  • Discussion of transfection methods, vectors, synthesis, and chemical modification strategies.

Main Results:

  • Hammerhead ribozymes demonstrate high specificity in cleaving target mRNAs.
  • Both endogenous and exogenous ribozyme systems offer distinct advantages and challenges for cellular delivery and function.
  • Successful inhibition of protein targets in cellular and animal models has been demonstrated.

Conclusions:

  • Hammerhead ribozymes are a promising tool for early drug target validation, offering specificity and efficiency.
  • Understanding ribozyme design and delivery is key to their successful application in pharmaceutical research.
  • Ribozyme technology provides a viable alternative to other gene silencing approaches for target validation.

Related Experiment Videos